A Study to Investigate the Safety, Tolerability, and Efficacy of SAR446268, an Adeno-associated Viral Vector-mediated Gene Therapy in Participants Aged 10 to 55 Years of Age With Non-congenital Myotonic Dystrophy Type 1

Recruiting now · Phase 1/Phase 2

Conditions studied: Myotonic Dystrophy

In brief

This is a Phase 1/Phase 2 open-label single arm, multicenter, and multinational study with SAR446268 for treatment of male and female participants 10 to 55 years old with non-congenital myotonic dystrophy (DM) type 1 (DM1). The purpose of this study is to evaluate the safety and efficacy of SAR446268 in knocking down dystrophia myotonica protein kinase (DMPK) messenger ribonucleic acid (mRNA) levels and improving neuromuscular function in DM1 participants receiving a single intravenous (IV) administration of SAR446268. The study consists of a dose escalation part (Part A) during which single ascending doses of SAR446268 will be evaluated in 3 distinct cohorts and an optional fourth dose cohort. Once a safe and effective dose is identified, additional participants will be treated in Part B, the dose expansion phase of the study. The study duration will be 112 weeks (approximately 2 years) for each participant in Parts A and B respectively and includes an optional pre-screening period, approximately 8-week screening phase and a 104-week follow-up period post-SAR446268 administration.

Key facts

Study ID: NCT06844214
Run by: Sanofi
People needed: 32
Starts: 2025-07-23
Expected to finish: 2032-04-27
Last updated by the study team: 2026-06-01

Who can join

Age: 10 and older, up to 55. Sex: any. Healthy volunteers: not accepted.

You may qualify if…

Participants are eligible to be included in the study only if all of the following criteria apply:
For Part A, participants must be 18 to 55 years of age inclusive, at the time of signing the informed consent.
For Part B, participants must be as follows:
10 to 17 years of age inclusive, at the time of signing the informed consent or,
18 to 55 years of age inclusive, at the time of signing the informed consent.
Participants with non-congenital onset DM1
Participants presenting with signs of DM1 including myotonia and muscle weakness, as diagnosed previously by a clinician based on medical history.
Participants with genetic diagnosis of DM1 [cytosine-thymine-guanine (CTG) repeat length ≥50 in one allele from medical history]
Participants who can walk independently for at least 10 meters at screening (orthoses and ankle braces allowed).

You may not qualify if…

Participants are excluded from the study if any of the following criteria apply:
Participants with neutralizing antibodies against the AAV.SAN011 capsid
Participants with left ventricular ejection fraction <50%
Participants with liver or biliary disease defined as having at least one of the following:
ALT >3 x ULN and AST >3 x ULN
Alkaline phosphatase >3 x ULN
Total bilirubin >1.5 x ULN (unless has a genetically confirmed diagnosis of Gilbert's syndrome)
Direct bilirubin ≥1.5 x ULN
Participants with International normalized ratio >1.5
Participants with renal disease defined as:
Serum creatinine >1.5 x ULN and/or estimated glomerular filtration rate <60 mL/min/1.73 m2 as determined by Chronic Kidney Disease Epidemiology Collaboration (2021) for those age ≥18 years and Bedside Schwartz Equation for those <18 years
Participants with chronic respiratory insufficiency and on long term/hull-time ventilatory assistance requiring at least 6 hours per day for at least 21 consecutive days.
Participants with contraindication to corticosteroid or with conditions that could worsen in the presence of corticosteroids, as determined by the Investigator.
Participants with active hepatitis B or C infection; HBsAg (+), or HCV RNA (+), or current antiviral therapy for either.
Participants with HBcAb (+) who are not amenable for prophylactic anti-HBV therapy or pre-emptive therapy guided by serial HBV DNA monitoring during the corticosteroids therapy.
Participants at high risk for tuberculosis reactivation during the corticosteroids therapy as determined by the Investigator.
Participants with a known HIV infection
Participants with serious intercurrent illness that, in the opinion of the Investigator, would preclude participation in the study or potentially decrease survival.
Participants with recent history of or current drug or alcohol abuse in the past 12 months prior to screening.
Participants with history of tibialis anterior biopsy within 12 weeks from Day 1 or planning to undergo tibialis anterior biopsies during the duration of this clinical trial.
Participants with significant developmental delay, intellectual disability, or behavioral neuropsychiatric manifestations as determined by the Investigator.
Participants with previous systemic corticosteroids treatment at doses of >5 mg/day within 15 days of Day 1
Participants with previous treatment with anti-myotonic medication within 15 days of Day 1
Participants not suitable for participation, whatever the reason, as judged by the Investigator, including medical or clinical conditions, or participants potentially at risk of noncompliance to study procedures.
Participants who have been classified as severe cardiac risk by the Investigator.

Where it is running

University of Florida, 2004 Mowry Road - Site Number: 8400005 — Gainesville, Florida, United States (enrolling)
University of South Florida - Neuromuscular Research, 13330 USF Laurel Drive - Site Number: 8400001 — Tampa, Florida, United States (enrolling)
Columbia University Medical Center - Neurological Institute, 710 W. 168th, 2nd floor, suite 204 - Site Number : 8400003 — New York, New York, United States (enrolling)
Virginia Commonwealth University Medical Center- Site Number : 8400006 — Richmond, Virginia, United States (enrolling)
Hospital Italiano de Buenos Aires, Juan Domingo Peron 4190 - Site Number: 0320001 — Buenos Aires, Argentina (enrolling)
Investigational Site Number : 0360001 — Brisbane, Queensland, Australia (enrolling)
The Montreal Neurological Institute and Hospital, 3801 rue University - Site Number: 1240001 — Montreal, Quebec, Canada (enrolling)
Investigational Site Number : 3760002 — Ramat Gan, Israel (enrolling)
Investigational Site Number : 8260002 — Newcastle upon Tyne, United Kingdom (enrolling)

Full record on ClinicalTrials.gov

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